Therapy for paroxysmal nocturnal hemoglobinuria (PNH) is evolving rapidly, spurred by the availability of biologic therapies that target the underlying hemolytic defect or the abnormal hematopoietic stem cell. However, many questions remain regarding when to initiate these costly therapies and how to balance therapy for symptoms of hemolysis with therapy for bone marrow failure.

If not obtained as part of the initial diagnostic evaluation, all patients should have baseline testing that includes a complete blood count (CBC) with differential, reticulocyte count, lactate dehydrogenase (LDH), biochemical profile, and flow cytometry for PNH. We also obtain iron studies (serum iron, total iron binding capacity, and ferritin) and a D-dimer level.

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